MC-DMD - Metformin and L-Citrulline

MC-DMD - Metformin and L-Citrulline in Duchenne muscular dystrophy

Dirk Fischer
University Children’s Hospital, Basel, Switzerland

Wednesday 25th October 2017

This is a proposal from Dr. Dirk Fischer and colleagues at the University Children’s Hospital in Basel to develop metformin and L-citrulline in combination as a therapy for Duchenne muscular dystrophy. The proposal aims to assess safety, clinical endpoints and biomarkers in a cohort of ambulatory boys with DMD.

TACT welcomed the fact that previous clinical trials seem to support the rationale and also appreciated the previous experience of the investigator. The committee felt that the planned study was well designed and grounded not only in the investigators’ experience but also the published literature on DMD trials and outcome measures.

The report makes recommendations about the mode and timing of drug delivery, a dose-ranging study to look at each drug separately, use of existing biomarker data and statistical analysis, in addition to some further pre-clinical animal work.

To request a full copy of the report please contact the applicant: Dirk Fischer

 
20 Dec 2017